Severe sialorrhoea and neurodisabilties in children
Conditions
Interventions
Trade Name: Sialanar
Product Name: Sialanar
Pharmaceutical Form: Oral solution
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Buccal use
Sponsors
Proveca Pharma LTD
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Children aged = 3 years old and =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Children unwilling to provide assent to participate in the study. (children who are unable to provide assent should be considered eligible). 2. Botulinum injection for sialorrhoea given within 6 months of enrolment. 3. Any anticholinergic therapy used in the previous 4 weeks. 4. Scopoderm patch used in the previous 4 weeks. 5. History of surgery for drooling in the previous 12 months. 6. Children prescribed non-permitted concomitant medication as defined in section 7.2.2 7. Children in whom anticholinergics are contraindicated such as those with glaucoma, myasthenia gravis, urinary retention, severe renal impairment, history of intestinal obstruction, ulcerative colitis, paralytic ileus, pyloric stenosis or hypersensitivity to the active substance or the excipient. 8. On-going or programmed orthodontic treatment over the study period. 9. Untreated oro-mandibular dystonia (isolated lingual dystonia accepted), clinical gastro oesophageal reflux, dental inflammatory condition (dental caries, gingivitis…). 10. Family and carers unable to commit to the schedule of the study protocol. 11. Female patients who are lacting or pregnant 12. Female patients who are planning a pregnancy within the study period 13. Patients having participated in another clinical study within at least 30 days or within 5 half-lives of last dose of IMP (whichever is longer).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To measure the efficacy of Sialanar® when used in addition to standard of care in France for children with severe sialorrhoea related to chronic neurodisabilities compared to placebo plus standard of care (SOC).;Secondary Objective: To measure the quality of life in relation to drooling of Sialanar® when used in addition to SOC in France compared to placebo plus SOC for children with severe sialorrhoea. To assess the tolerability of Sialanar® when used in addition to SOC in France for children with severe sialorrhoea ;Primary end point(s): The Change in Drooling Impact Scale (DIS) between baseline and D84. The DIS scale is a questionnaire of 10 questions with a score of 100 points which is completed by the patient's entourage (family and/or caregivers). This scale has a good validity and sensitivity to changes in the context of the evaluation of the drooling in children with CP, with a score difference of 13.6 points considered as clinically relevant (Reid 2010). ;Timepoint(s) of evaluation of this end point: The evaluation of the primary efficacy endpoint will be performed for the full analysis set (ITT) and for the mITT analysis set. The primary analysis set will be the full analysis set. All statistical tests will be two-sided and the significance level will be 5%. Mean difference in DIS between baseline and D84 (+/- 5 days) between the Sialanar and placebo group. A 13.6 points change in DIS Scale score will be considered as a clinically relevant change. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Other efficacy endpoints: 2. Change in DIS Scale between baseline and D28. 3. Proportion of responders at D84 (a response is defined as a DIS improvement = 13.6 points). 4. Proportion of responders at D28 (a response is defined as a DIS improvement = 13.6 points). 5. Proportion of good responders at D84 (a good response is defined as a DIS improvement = 28 points). 6. Changes in the number of used bibs or clothing over 7 days (Item 3 of the DIS) at D84 7. Changes in the number of used bibs or clothing over 7 days (Item 3 of the DIS) at D28 Quality of life endpoints: - Change in the DIS 10-point item 9 from baseline. “To what extent did your child’s drooling affect his or her life?” to D28 and D84. - Change in in the DIS 10-point item 10 from baseline. “To what extent did your child’s dribbling affect you and your family’s life?” to D28 and D84. - The change in DISABKIDS instrument from baseline to D84. Safety Endpoints: - Adverse events recorded from baseline to day 84, including: - all AE, - all AE with the exception of dose dependent expected AE related to titration, - all SAE. - AEs leading to discontinuation of study medication - all treatment-related AEs - all treatment-related AEs with the exception of dose dependent expected AE related to titration Adverse events will be collected at every visit from parent or carer and participant where possible. All adverse events will be recorded at every scheduled visit plus outside of visits as required. Open- label study extension endpoints: - Changes in DIS Scale between baseline and D252 and between D84 and D252 in the previous ‘Sialanar®-arm’ patients. - Change in DIS Scale between D84 and D252 for patients previously taking placebo - Change in the DIS 10-point item 9. “To what extent did your child’s drooling affect his or her life?” between baseline and D252. - Change in the DIS 10-point item 10. “To what extent did your child’s dribbling affect you and your family’s life?” between b | — |
Countries
France
Contacts
Public ContactCRO
Kappa Santé
Outcome results
None listed